I was the president of Pfizer Global Research and Development in 2007 where I managed more than 13,000 scientists and professionals in the United States, Europe, and Asia. I've received numerous awards including an Honorary Doctor of Science degree from the University of New Hampshire. I am also the author of "Drug Truths: Dispelling The Myths Of R&D" and the recently published Devalued And Detrusted: Can The Pharmaceutical Industry Restore Its Broken Image?" I am also a senior partner at PureTech Ventures.

Should the Cystic Fibrosis Foundation Invest $58 Million in Pfizer Research?

As National Institutes of Health (NIH) funding has stagnated over the past few years, an increasingly important source of research funding for academic laboratories and start-up biotech companies has been non-profit organizations like the Michael J. Fox Foundation and the American Cancer Society. Researchers with exciting, cutting edge, but unproven, projects often get their start from grants funded by non-profits. As these projects make interesting progress, it is not unusual for further funding to be found from venture capital firms, biotech companies or even big pharma, thereby allowing these programs to move toward fully fledged R&D projects. The role of non-profit organizations in the early stage eco-system of the discovery of new drugs has become quite important.

“We are excited to expand our efforts with Pfizer to accelerate the development of more therapies that treat the root cause of CF and benefit the greatest number of people with the disease. Pfizer brings impressive technical and scientific expertise, along with its commitment to improving the lives of people with cystic fibrosis.”

Like many big pharma companies, Pfizer (PFE) had avoided rare disease R&D for many years, and thus this area was dominated by biotech companies such as Genzyme. However, the commercial success of drugs for rare diseases such as Genzyme’s Cerezyme with sales of over $700 million annually, led companies like Pfizer and GlaxoSmithKline (GSK) to form their own research units in rare diseases. Genzyme itself was acquired by Sanofi as part of the latter’s expansion into this field. One of Pfizer’s first steps in 2010 was to buy FoldRx, a small company that focused on developing treatments for diseases caused by the improper folding of proteins. In fact, when Pfizer purchased FoldRx, it inherited a partnership that the latter had with the CFF.

“We want to know, especially from Big Pharma, that some might see as interlopers seeking to bleed the last of high profits from a new area now that their more common drugs are going generic, that entry into this area is not a fleeting thought of profits.”

Pfizer made a firm decision to enter the world of rare diseases. By buying FoldRx, it assumed its research capability as well as FoldRx’s other commitments, such as working with the CFF. Theoretically, Pfizer must have assumed that it would need to commit resources to prosecuting the FoldRx portfolio. Thus, I am very surprised that Pfizer is making the CFF essentially pay for the early discovery work to find CF drug candidates. Would Pfizer have NOT done this research without the CFF funding? If the answer is yes, then one must challenge Pfizer’s commitment to rare diseases.

My view is that if Pfizer is truly committed to rare disease R&D, it should have been doing this work anyway. Clearly, it didn’t think it important enough to fund internally. By requiring the CFF to pay for it, Pfizer is diverting precious research funds that the CFF could be using to fund numerous other programs in universities, research institutes,and small start-ups companies. In the life sciences eco-system, $58 million can go a long way. Should the world’s largest pharmaceutical company have its hand out to the CFF? Couldn’t Pfizer have found the <$10 million/year needed to fund this research? If not, one has to question the level of commitment that exists for rare disease R&D in this company.

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John, thanks again for getting people talking about this. Interesting Twitter dialogue in last few days between @oli_rayner and @sciencescanner about funding the development of new drugs for CF and other orphan diseases culminating in a must-read post today by David Grainger on his DrugBaron blog: http://bit.ly/TG4uJn Situation with Kalydeco in the UK is acute but David’s blog sets out a clear intellectual framework for discussion on how to address the economics of orphan drug development more globally. Oli